Gene therapy offers ray of hope for the blind.

May 15 2003.

By Patrick Leeman,
The Mercury.

New hope: Ground-breaking research into retinal gene therapy has given new hope to people with blindness in their families. One of the pioneers of this therapy is Professor William Hauswirth, who is based at the department of ophthalmology at the University of Florida. He is attending a genetics conference in Durban.

Researchers in the United States have made dramatic strides in finding a treatment for blindness through retinal gene therapy.

The therapy, which involves the injection under the retina of beneficial human gene material to restore what was missing in the defective gene, has proved extremely effective when applied to dogs.

Now geneticists and scientists in the US are waiting for the go-ahead to use human volunteers for such trials.

One of the pioneers of this ground-breaking research, Professor William Hauswirth, said this week that the results of the studies held enormous potential for the millions of blind and partially sighted people all over the world.

Every one of the 30 blind dogs responded positively
Hauswirth said that every one of the 30 originally blind dogs in the study had responded positively to the treatment. In some cases the dogs had regained 20 percent of their sight.

It was possible, if further tests went according to plan, that the first human could be treated by 2006.

Hauswirth said the treatment had application for a wide range of retinal conditions.

It could be used to treat Retinitis Pigmentosa, a condition causing night blindness and then a gradual loss of side vision, leading to tunnel vision.

Other causes of blindness, he said, were macular degeneration, which was related to age, diabetic retinothopy (blindness caused by diabetes), and glaucoma.

Hauswirth said there had been spectacular strides in retinal gene therapy since 1990.

At that time it was estimated that there were 180 genes which caused blindness. Researchers had now identified half of them.

Although the research involving the dogs was aimed at a specific gene condition, the results had implications for all therapies concerned with retinal disease.

Hauswirth said sight had been restored in the dogs after an average of three months. Their vision had remained stable for three years.